Shares of Biogen Inc. (NASDAQ: BIIB) and, especially, Ionis Pharmaceuticals Inc. (NASDAQ: IONS) made handy gains early Monday morning following favorable interim results from a late-stage trial. The companies announced that nusinersen, their investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint prespecified for the interim analysis of ENDEAR, the Phase 3 trial evaluating nusinersen in infantile-onset (consistent with Type 1) SMA.
Ultimately, the analysis found that infants receiving nusinersen experienced a statistically significant improvement in the achievement of motor milestones compared to those who did not receive treatment. Nusinersen demonstrated an acceptable safety profile in the trial. As a result of these findings, Biogen has exercised its option to develop and commercialize nusinersen globally and paid Ionis a $75 million license fee. Biogen will initiate regulatory filings globally in the coming months.
The other studies in the nusinersen program, which include CHERISH (later-onset consistent with Type 2) and NURTURE (pre-symptomatic infants), will continue as planned. The goal for these studies is to collect the data to demonstrate the safety and efficacy of nusinersen in these populations.
In conclusion, Biogen is now responsible for all nusinersen development, regulatory and commercialization activities and costs. Ionis will complete the Phase 3 studies and work with Biogen on regulatory filings. The two companies also will work together to transition the clinical programs that Ionis is conducting to Biogen. Ionis is eligible to receive tiered royalties on any potential sales of nusineren up to a percentage in the mid-teens, in addition to up to $150 million in milestone payments based on regulatory approvals.
B. Lynne Parshall, chief operating officer of Ionis, commented:
We are hopeful that nusinersen, if approved, will make a meaningful difference in the lives of patients and families affected by SMA. We look forward to working with Biogen on completing the clinical program and preparing for what we hope is a positive regulatory review. Nusinersen is the first antisense drug from our neurological disease franchise to advance to regulatory review, and it illustrates the potential of our antisense technology to address severe diseases that other therapeutic modalities are unable to address adequately.
Shares of Biogen closed Friday at $289.93, with a consensus analyst price target of $332.50 and a 52-week trading range of $223.02 to $338.51. Following the announcement, the stock was up about 4.2% at $302.00 in early trading indications Monday.
Ionis shares closed Friday at $29.19, within a 52-week range of $19.59 to $62.68 and with a consensus price target of $32.38, In Monday’s premarket, the stock was up nearly 40% at $40.61.